Thiocyanate is a molecule found throughout the human body. Recent studies in animals have shown that thiocynate could be used as a treatment for lung inflammation, although more research is needed. These new insights are particularly important for the CF community, as thiocynate could potentially be useful as a treatment for…
News
Last week, the Her Royal Highness the Countess of Wessex, Sophie Rhys-Jones, officially inaugurated the Wolfson Cystic Fibrosis Center at Nottingham City Hospital. During the opening day, HRH made time to meet with CF patients, as well as the center’s staff and key donors. The new 250-bed center is estimated to be…
Gene therapy in the context of treating cystic fibrosis made a significant step forward with the announcement of positive data from a Phase IIb clinical trial investigating the VGXI-manufactured aerosolized DNA plasmid expressing Cystic Fibrosis Transmembrane Conductance Receptor (CFTR) — an experimental CF therapy that improved a key lung function…
Spyryx Biosciences, Inc., a private bio-pharmaceutical firm advancing new therapeutics to address obstructive lung diseases, recently announced it has received an award from Cystic Fibrosis Foundation Therapeutics that will provide support to the development of Spyryx’s therapeutic peptides to address cystic fibrosis. “We appreciate the generous support of the Cystic Fibrosis…
A study recently published in the journal The Lancet Respiratory Medicine revealed encouraging results of a phase 2b clinical trial assessing the safety and efficacy of a non-viral gene therapy based on a gene defective in cystic fibrosis (CF) patients. The study is entitled “Repeated…
A new study revealed that microRNA16 — a small non-coding RNA genes that regulates gene expression — can restore the F508del-CFTR protein function in airway cell lines and primary cultures of differentiated human bronchial epithelia from F508del homozygotes that express mutant CFTR protein endogenously. The finding is significant, as…
Researchers at the University of Arkansas for Medical Sciences and the Department of Health, Arkansas reported a rare case of pulmonary tuberculosis in a cystic fibrosis (CF) patient. The report, which was published in the North American Journal of Medical Sciences and is entitled “…
The U.S. Food and Drug Administration on Thursday, July 2 announced that it has approved the first cystic fibrosis treatment that targets the cause of the disease. Orkambi is an oral ivacaftor/lumacaftor combination medication for treatment of CF that targets the underlying cause of the disease in people who have…
Researchers at Stanford University School of Medicine recently reported that Hispanic patients with cystic fibrosis (CF) have a higher mortality rate than non-Hispanic patients. The study was published in the journal Chest and is entitled “Assessing differences in mortality rates and risk factors…
Accuracy and efficiency are of utmost importance when deciding on a diagnostic tool to detect cystic fibrosis in newborns. The gold standard of detection is measuring sweat ion concentration, as patients with defect CFTR proteins have an elevated electrolyte content in their sweat. However, another method that is now only…
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